The grants are part of The Target ALS Diagnosis Initiative, a $15 million comprehensive effort to discover ALS biomarkers. “Biomarker” is a broad term that refers to any biological substance that can be objectively measured, giving reliable insight into a disease. Biomarkers are important for tracking disease progression, and in establishing accurate prognoses.
“Listening to what our scientific community needs to advance their work, and responding with innovative approaches and resources is how we help exponentially advance ALS research,” Manish Raisinghani, PhD, CEO of Target ALS, said in a press release.
“We are now at a juncture where reliable biomarkers are an increasingly acute need. We look forward to the contribution of these consortia in this search, which could greatly improve the lives of ALS patients,” Raisinghani added.
These projects will be led by a collaborative team of researchers from both academia and the pharma/biotech industry. Such collaborations were an award requirement.
One project, conducted by investigators from Biogen and Washington University, aims to characterize variants of the protein neurofilament light chain (NfL) as potential ALS biomarkers.
NfL is commonly used as a general marker of nerve cell damage. However, conventional methods for measuring NfL aren’t able to differentiate between different forms of this protein. This project aims to use mass spectrometry, a highly sensitive technique that can make such differentiation possible, to analyze NfL in people with and without ALS. The goal is to find disease-relevant NfL variations.
Another project, a collaboration between researchers from NeuroDex, Massachusetts General Hospital, and Johns Hopkins University, will investigate extracellular vesicles (EVs) as potential ALS biomarkers.
EVs are essentially little packets of cellular material, such as protein and RNA, wrapped in a membrane. Cells release EVs for a variety of biological functions; the exact contents of any given EV depends on its cell of origin and other factors.
Researchers here have already demonstrated that the protein cargo in EVs from neurons (nerve cells) is altered in people with ALS. Now, they aim to expand and finalize this biomarker signature, test it in more people, and investigate its association with several of the disease’s neurological features.
The remaining two projects are investigating TDP-43, a protein whose accumulation in the brain in abnormal forms is a hallmark of ALS.
Disease-associated TDP-43 can easily be detected in samples of postmortem brain tissue, but it cannot be accurately measured in living people. Researchers at AC Immune and Massachusetts General Hospital will work to develop tests that can measure TDP-43 in fluid samples, like blood, taken from patients. These tests are being developed using antibodies that specifically bind to TDP-43.
“This award provides further validation for our comprehensive diagnostic and therapeutic programs targeting pathological TDP-43,” Andrea Pfeifer, CEO of AC Immune, said in a company press release.
Researchers at Biogen and Johns Hopkins will also work to better understand how other proteins are associated with signaling pathways related to TDP-43. In addition to advancing an understanding of the molecular underpinnings of ALS, these associated proteins may serve as biomarkers themselves.
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